Blood Research Institute, Versiti, Milwaukee, WI, USA; Department of Cell Biology, Neurobiology, and Anatomy, Medical College of Wisconsin, Milwaukee, WI, USA.
Blood Research Institute, Versiti, Milwaukee, WI, USA.
Neoplasia. 2021 Mar;23(3):337-347. doi: 10.1016/j.neo.2021.01.003. Epub 2021 Feb 20.
Acute myeloid leukemia (AML) affects tens of thousands of patients a year, yet survival rates are as low as 25% in certain populations. This poor survival rate is partially due to the vast genetic diversity of the disease. Rarely do 2 patients with AML have the same mutational profile, which makes the development of targeted therapies particularly challenging. However, a set of recurrent mutations in chromatin modifiers have been identified in many patients, including mutations in the cohesin complex, which have been identified in up to 20% of cases. Interestingly, the canonical function of the cohesin complex in establishing sister chromatid cohesin during mitosis is unlikely to be the affected role in leukemogenesis. Instead, the cohesin complex's role in DNA looping and gene regulation likely facilitates disease. The epigenetic mechanisms by which cohesin complex mutations promote leukemia are not completely elucidated, but alterations of enhancer-promoter interactions and differential histone modifications have been shown to drive oncogenic gene expression changes. Such changes commonly include HoxA upregulation, which may represent a common pathway that could be therapeutically targeted. As cohesin mutations rarely occur alone, examining the impact of common co-occurring mutations, including those in NPM1, the core-binding factor complex, FLT3, and ASXL1, will yield additional insight. While further study of these mutational interactions is required, current research suggests that the use of combinatorial genetics could be the key to uncovering new targets, allowing for the treatment of AML patients based on their individual genetic profiles.
急性髓系白血病(AML)每年影响成千上万的患者,然而在某些人群中的存活率低至 25%。这种低存活率部分归因于该疾病的巨大遗传多样性。很少有两名 AML 患者具有相同的突变谱,这使得靶向治疗的开发特别具有挑战性。然而,在许多患者中已经确定了一组染色质修饰物的反复突变,包括在多达 20%的病例中发现的黏合蛋白复合物中的突变。有趣的是,黏合蛋白复合物在有丝分裂过程中建立姐妹染色单体黏合的经典功能不太可能是白血病发生中的受影响作用。相反,黏合蛋白复合物在 DNA 环化和基因调控中的作用可能促进疾病。黏合蛋白复合物突变促进白血病的表观遗传机制尚未完全阐明,但已表明增强子-启动子相互作用的改变和差异组蛋白修饰可驱动致癌基因表达变化。这种变化通常包括 HoxA 的上调,这可能代表一种可治疗靶向的共同途径。由于黏合蛋白突变很少单独发生,因此研究常见的共同发生的突变(包括 NPM1、核心结合因子复合物、FLT3 和 ASXL1 的突变)将提供更多的见解。虽然需要进一步研究这些突变相互作用,但目前的研究表明,组合遗传学的使用可能是揭示新靶标的关键,可根据患者的个体遗传特征治疗 AML 患者。